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            Lead Product(s): Arimoclomol

            Therapeutic Area: Neurology Product Name: Miplyffa

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 07, 2021

            Details:

            Orphazyme announced that ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.

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            Lead Product(s): Arimoclomol

            Therapeutic Area: Neurology Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 22, 2020

            Details:

            Orphazyme has received Fast Track Designation from the US Food and Drug Administration (FDA) for the development of arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS).